Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial (NCT02137850) involved children with haemophilia A — a condition where the blood does not clot properly due to low levels of a protein called Factor VIII. The trial looked at a treatment called N8-GP (also known as turoctocog alfa pegol), which is a modified form of that clotting protein. Participants were placed into one of three groups depending on their treatment stage: a pre-prophylaxis group (55 children started, 44 completed), a prophylaxis group — meaning regular preventive treatment — (69 children started the main phase, 55 completed it, and 49 then completed an extension phase), and a small immune tolerance induction group (8 children started, 4 completed). This last group was for children whose immune systems had started producing antibodies that block the clotting protein. The reported data shows that the primary thing being measured was how many children developed inhibitory antibodies — that is, antibodies that block the clotting protein from working. According to the results reported on ClinicalTrials.gov, 11 children in the pre-prophylaxis group and 10 children in the prophylaxis group developed these antibodies. Of those, 3 in the pre-prophylaxis group and 8 in the prophylaxis group had what are described as "high titre" inhibitors, meaning the antibody levels were above a particular threshold (5 Bethesda Units). For the prophylaxis group, the reported data shows an average of approximately 1.35 bleeding episodes per patient per year while on regular preventive treatment, and an average dose of 68.9 IU/kg was used for prophylaxis. The reported data also shows the number of adverse events (unexpected or unwanted medical occurrences during the trial) recorded across the groups. In the pre-prophylaxis group, 116 adverse events were recorded in total, including 24 classified as serious and 17 as events of special interest. In the prophylaxis group, 644 adverse events were recorded in total, including 56 serious and 47 of special interest. In the immune tolerance induction group, 32 adverse events were recorded in total, including 3 serious and none of special interest. The trial also tracked how well bleeding episodes were controlled using a four-point scale (excellent, good, moderate, or none) — the breakdown of individual bleeding episodes across these categories was recorded, but a single overall summary figure was not provided in the reported data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 37597724) ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 3 Haemophilia Trial, Completed
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- A parent or guardian must give their consent before any trial-related activities take place
- The patient must be a boy under 6 years old at the time consent is signed
- The patient must have been diagnosed with severe haemophilia A (meaning their clotting factor VIII level is 1% or below), confirmed by medical records or lab results
- The patient must not have previously used purified clotting factor products (having had up to 5 exposures to blood products such as transfusions is acceptable)
Who may not be able to join:
- The patient has any history of developing inhibitors (antibodies) against factor VIII, as shown in medical records
- The patient has a known or suspected allergy to the trial product or similar products
- The patient has previously taken part in this same trial and received a dose of the trial product
- The patient has received any other experimental medicine or treatment within 30 days before the screening visit
- The patient has any other clotting disorder, either one they were born with or one they developed, besides haemophilia A
- The patient has any long-term or serious health condition that, in the doctor's judgement, could put them at risk or make it difficult to follow the trial requirements
- The patient's parent(s) or legal guardian is unable or unwilling to cooperate with the trial, or there is a language barrier that prevents them from fully understanding what the trial involves
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Global Clinical Registry (GCR, 1452), Novo Nordisk A/S
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
2 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII)
Can't join this trial?
Data last synced from ClinicalTrials.gov: 23 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.